39 - Metabolic Conditions

Abstract

Liver transplantation is now very successful, with more than 85% long-term survival into adult life. When considering the impact of liver transplantation for metabolic disease, two independent factors need to be considered—whether or not the defect causes significant liver disease and whether or not it is confined to the liver. When considering transplantation, many factors need to be considered, including the local success of transplantation, the impact of the metabolic disease on the patient and family, and the potential for future therapeutic developments. When transplantation is undertaken for a liver-based defect, there is a lifelong complete correction of the defect. A residual extrahepatic defect will have an impact on the outcome of liver transplantation, and the severity of this defect must be considered as part of the transplantation assessment process. Access to a multidisciplinary team with expertise in metabolic disease, liver disease, and other relevant organ-based specialties is crucial. Most children will receive a transplant from a cadaveric donor, but living related transplantation from a heterozygote parent is usually safe and effective. Auxiliary liver transplantation has a small but useful role when partial correction of the defect is helpful, and there is a future prospect of gene therapy.